Identification of Novel Genetic Modifiers of Glucocerebrosidase Activity as Targets fro Parkinson's Other Degenerative Diseases

Project: Research project

Project Details

Description

An abundance of genetic, experimental, and clinical evidence has implicated reduced activity of the enzyme glucocerebrosidase (GCase) in the onset of Parkinson’s disease (PD). As a result, increasing enzymatic activity of GCase is considered a promising therapeutic strategy to treat patients with PD. The goal of this collaboration is to identify novel modifiers that play a role in regulating GCase activity within the cell. These modifiers will improve our understanding of PD pathogenesis by identifying proteins and pathways involved in regulating the activity of lysosomal enzymes. Identification of druggable pathways involved in regulating GCase activity could reveal attractive new therapeutic targets for disease modification in PD. While Aim 1a (see below) is a higher priority, we feel that Aim 1b could yield valuable and likely complimentary information regarding physiological differences in dopaminergic neurons from affected and unaffected family members.
StatusFinished
Effective start/end date12/20/1812/19/21

Funding

  • AbbVie Inc. (Agmt 12/20/18)

Fingerprint

Explore the research topics touched on by this project. These labels are generated based on the underlying awards/grants. Together they form a unique fingerprint.